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Orphanet Journal of Rare Diseases|February 23, 2022
Collaborative research protocol to define patient-reported experience measures of the cystic fibrosis care pathway in France: the ExPaParM studyD Pougheon Bertrand, A Fanchini, P Lombrail, et al.
Orphanet Journal of Rare Diseases|February 23, 2022
Understanding disease symptoms and impacts and producing qualitatively-derived severity stages for MPS IIIA: a mixed methods approachSally Lanar, Samantha Parker, Cara O'Neill, et al.
Orphanet Journal of Rare Diseases|February 17, 2022
Risk factors for pregnancy-related clinical outcome in myasthenia gravis: a systemic review and meta-analysisManqiqige Su, Xiaoqing Liu, Liang Wang, et al.
Orphanet Journal of Rare Diseases|April 2, 2022
Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS)Lina Titievsky, Tilman Schuster, Ronnie Wang, et al.
Orphanet Journal of Rare Diseases|April 8, 2022
Health utilities and costs for neuromyelitis optica spectrum disorderDyfrig A Hughes, Siobhan Bourke, Angela Jones, et al.
Orphanet Journal of Rare Diseases|March 22, 2022
Systematic estimation of cystic fibrosis prevalence in Chinese and genetic spectrum comparison to CaucasiansQi Ni, Xiang Chen, Ping Zhang, et al.
Orphanet Journal of Rare Diseases|March 25, 2022
Analysis of the relationship between phenotypes and genotypes in 60 Chinese patients with propionic acidemia: a fourteen-year experience at a tertiary hospitalYi Liu, Zhehui Chen, Hui Dong, et al.
Orphanet Journal of Rare Diseases|March 29, 2022
Qualitative interviews with adults with Classic Galactosemia and their caregivers: disease burden and challenges with daily livingJason A Randall, Carolyn Sutter, Stella Wang, et al.
Orphanet Journal of Rare Diseases|March 29, 2022
Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative studyMichaël Acquadro, Alexia Marrel, Maria A Manso-Silván, et al.
Orphanet Journal of Rare Diseases|March 30, 2022
Survival of children with rare structural congenital anomalies: a multi-registry cohort studyAlessio Coi, Michele Santoro, Anna Pierini, et al.
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